A supplemental new drug application (sNDA) was submitted to the Food and Drug Administration (FDA) for cannabidiol (Epidiolex, GW Pharmaceuticals, Carlsbad, CA). The sNDA seeks to expand the cannabidiol label to include the treatment of seizures associated with tuberous sclerosis complex (TSC), a rare genetic condition. Cannabidiol is currently approved to treat seizures associated with Lennox-Gastaut syndrome (LGS) or Dravet syndrome (DS) and has been granted orphan drug designation from the FDA for the treatment of TSC.
The sNDA is supported by data from a phase 3 safety and efficacy study, results of which were recently presented at the American Epilepsy Society 2019 annual meeting. The study met its primary endpoint, with participants treated with cannabidiol 25 mg/kg/day experiencing a significantly greater reduction from baseline in TSC-associated seizures compared with placebo (49% vs 27%; P=.0009). Results for the 50 mg/kg/day dose group were similar, with seizure reductions of 48% from baseline vs 26.5% for placebo (P=.0018). All key secondary endpoints were supportive of the effects on the primary endpoint. The safety profile observed was consistent with findings from previous studies, with no new safety risks identified.
"The submission of this sNDA for Epidiolex is an important step towards the prospect of offering a new treatment option for those patients with TSC who battle difficult-to-treat seizures," said CEO, Justin Gover. "Having already obtained approval for Epdiolex in the treatment of seizures associated with LGS and DS, this submission is based on positive phase 3 data showing that Epidiolex reduced TSC-associated seizures, which include both focal and generalized seizures types. We look forward to working with the FDA toward an expected approval later this year."
Bettina Balint, MD
Jason A. Ellis, MD; Benjamin W. Y. Lo, MD; Chirag G. Bhatia, BS; Yona Feit; Steven Mandel, MD; and Dana Shani, MD